Axovant announces recent progress in gene therapy programs
Axovant Gene Therapies announced recent progress in its gene therapy programs, including a 12-month update on its Parkinson's program. Major 2019 Accomplishments and Upcoming 2020 Milestones include: at 12 months post-dosing, patients in the first dose cohort demonstrated an average 22-point change from baseline in motor function as assessed by the UPDRS Part III "OFF" score, which represents a 37% improvement. Individual patient improvements were 24-points and 20-points, respectively. Reported data from the first child dosed with AXO-AAV-GM1 under an expanded access protocol, which suggested tolerability and clinical improvement between baseline and 6-months after vector administration with no reports of serious adverse events attributable to the gene therapy. In October 2019, at the European Society of Gene and Cell Therapy 27th Annual Congress, Dr. Terry Flotte presented evidence of clinical disease stabilization in two patients dosed with AXO-AAV-GM2 under an investigator-initiated IND. Initial observations suggested that AXO-AAV-GM2 was generally well-tolerated and associated with improvement in bioactivity outcomes. In addition, data suggested attainment of normal neurodevelopmental milestones and improvement in myelination on brain MRI.